Diagnosis and Therapy of Myelodysplastic Syndromes in Germany: A Retrospective Multicenter Analysis

Abstract

Background: Myelodysplastic syndromes (MDS) are a heterogenous group of clonal hematopoietic stem cell disorders. Patients and Methods: In order to assess current diagnosis and treatment patterns in Germany, the data of 269 patients with MDS from 57 representative centers were analyzed. Results: The most common symptom leading to an initial diagnosis of MDS was anemia (79%). WHO classification, cytogenetic analysis, and IPSS scoring were performed in 92, 67, and 61% of patients, respectively. 5q deletions were identified in 34% of patients whose cytogenetic status was analyzed. Symptomatic anemia was the major trigger for initiating therapy. 49% of patients received supportive care only, and 49% received active therapy (i.e., chemo-, immunomodulatory, or epigenetic therapy), including 5% who received allogeneic transplantation. Of those patients treated with active therapy, approximately half of the higher-risk patients received azacitidine, and approximately half of the lower-risk patients received lenalidomide. Overall, 80% of patients received some form of supportive care, mainly red blood cell transfusions. Conclusion: While the WHO classification system is widely used in clinical practice, karyotyping and IPSS risk assessment do not seem to be common standard. Despite encouraging data on the use of effective and novel drugs, such as lenalidomide and azacitidine in MDS therapies, management of the disease could be further improved by more widespread use of risk stratification of patients using cytogenetics and IPSS assessment.

Factors predicting early mortality after new diagnosis of myelodysplastic syndrome: A population‐based study

“…One strength of our study was the high degree of diagnostic certainty through pathologic evaluation, due to a rigorous central review process. Further, because we utilized direct chart review of treatment strategy and independent calculation of risk scores, we effectively eliminated the recall bias that can be seen with a survey‐based data collection approach as relied upon by several previously published studies . In addition to these strengths, several weaknesses must be considered.…”

Differences in community and academic practice patterns for newly diagnosed myelodysplastic syndromes (MDS) patients

“…Although various studies have evaluated the effectiveness of these agents [32-36], their use has declined with the growing evidence for improved disease modification with the hypomethylators and lenalidomide [37]. These differences may reflect a geographical divide, with two European studies showing higher utilization of alternative agents [15,16] compared to a 2008 US-based treatment strategy study [13] and the data presented here.…”

“…This is significantly consolidated compared to previous treatment reviews which documented use of androgens, immunosuppressive agents, hydroxyurea, and thalidomide [15,16]. Although various studies have evaluated the effectiveness of these agents [32-36], their use has declined with the growing evidence for improved disease modification with the hypomethylators and lenalidomide [37].…”

“…Numerous retrospective studies have evaluated disease and transplant-related factors for MDS transplants including age of recipient, pre-transplant disease burden, conditioning regimen intensity, and donor characteristics [38-41], yet the concern for high morbidity and mortality of this therapy combined with the generally older age of MDS patients has resulted in low utilization of this treatment strategy despite available data highlighting adequate tolerance in an older patient population [28]. The three main practice pattern studies to date reported transplant rates of only 2-5%, and all had a duration since diagnosis >1yr [13,15,16]. Our study was limited to one year follow-up since diagnosis, potentially underestimating the difference in transplant rates as more patients in our study may have been transplanted with continued observation past one year.…”

“…Additionally, independent calculation of risk scores and verification of treatment strategy by direct chart review effectively eliminated the recall bias that may accompany a survey-based approach to collection of treatment data as relied upon by several previously published studies [13-15]. The case accrual process worked to minimize selection bias by not limiting data collection to specified physicians, as in the US-based study, or to patients presenting for care within a narrowly-defined period (7 days, in the French study) [13,16].…”